Best Practice & Research Clinical Haematology
Volume 19, Issue 1 , Pages 35-49 , March 2006

Clinical uses of plasma and plasma fractions: plasma-derived products for hemophilias A and B, and for von Willebrand disease

  • Cassandra D. Josephson, MD (Assistant Director, Children's Healthcare of Atlanta Blood Banks and Transfusion Services; Assistant Professor, Pathology and Laboratory Medicine and Pediatrics)

      Affiliations

    • Corresponding Author InformationCorresponding author. Tel.: +1 404 785 4553; Fax: +1 404 785 1370.
  • Thomas C. Abshire, MD (Professor of Pediatrics and Director, Comprehensive Hemophilia Program)

References 

  1. Schimpf K. On the way to virus-safe blood coagulation factor concentrates. Semin Thromb Haemost. 2002;28:15–24
  2. Toole JJ, Knopf JL, Wozney JM, Sultzman LA, et al. Molecular cloning of a cDNA encoding human antihaemophilic factor. Nature. 1984;312:342–347
  3. Choo KH, Gould KG, Rees DJ, Brownlee GG. Molecular cloning of the gene for human anti-haemophilic factor IX. Nature. 1982;299(5879):178–180
  4. Rothschild C, et al. J Thromb Hemost Suppl. 2003;abstrOC215
  5. Auerswald G, Spranger Th, Brackmann HH. The role of plasma-derived factor VIII/von Willebrand factor concentrates in the treatment of hemophilia A. Haematologica. 2003;88:21–29
  6. Poon MC, Aledort LM, Anderle K, et al. Comparison of the recovery and half-life of a high purity factor IX concentrate with those of a factor IX complex concentrate. Factor IX Study Group. Transfusion. 1995;35:319–323
  7. Kavakli K, Gringeri A, Bader R, et al. Inhibitor development and substitution therapy in a developing country:Turkey. Haemophilia. 1998;4:104–108
  8. Srivastava A. Choice of factor concentrates for haemophilia: a developing world perspective. Haemophilia. 2001;7:117–122
  9. Lusher JM. Hemophilia A and B. In:  Lilleyman J,  Hann I,  Blanchette V editor. Pedeatric Hematology. 2nd edn. London, UK: Churchill Livingstone; 1999;p. 585–600
  10. Bona RD, Weinstein RA, Weisman ST, et al. The use of continuous infusion of factor concentrates in the treatment of hemophilia. Am J Hematol. 1990;
  11. Lane S. Successful transfusion of blood. Lancet. 1840;1:185–188
  12. Pool JG, Gershgold EJ, Pappenhagen AR. High-potency antihaemophilic factor concentrate prepared from cryoglobulin precipitate. Nature. 1964;18(203):312
  13. Hilgartner MW. The need for recombinant factor VIII: historical background and rationale. Semin Hematol. 1991;28(supplement 1):6–9
  14. Hoots WK. History of plasma-product safety. Transfus Med Rev. 2001;15:3–10
  15. Kasper CK, Lusher JM. Recent evolution of clotting factor concentrates for hemophilia A and B. Transfusion Practices Committee. Transfusion. 1993;33:422–434
  16. Farrugia A. Evolving perspectives in product safety for haemophilia. Haemophilia. 2002;8:236–243
  17. Accredo Health Inc. Announces Record Results; Third Quarter Earnings Increase 75%. Accessed 5-2-2004 at http://www.accredohealth.net/ahi/news/pr010430.html
  18. Factor VIII Product Shortage. World Federation of Hemophilia Information Update. 2001. Accessed on 4-2-04. at http://www.wfh.org/Content_Documents/Newsletters/IU_April01_Eng.pdf
  19. Soucie JM, Richardson LC, Evatt BE, et al. Risk factors for infection with HBV and HCV in a large cohort of hemophiliac males. Transfusion. 2001;41:338–343
  20. Chorba TL, Holman RC, Clarke MJ, et al. Effects of HIV infection on age and cause of death for persons with hemophilia A in the United States. Am J Hematol. 2001;66:229–240
  21. Fricke W, Augustyniak L, Lawrence D, Brownstein A, et al. Human immunodeficiency virus infection due to clotting factor concentrates: results of the Seroconversion Surveillance Project. Transfusion. 1992;32:707–709
  22. Groener A, Mehdi S, Schafer W, Spyra M. Human parvovirus B19 is inactivated by pasteurization, a dedicated virus reduction step in the manufacturing procedure of plasma derivatives [abstract]. Blood. 2003;102:4241
  23. Mannucci PM. Treatment of hemophilia: recombinant factor only? No. J Thromb Haemost. 2003;1:216–217
  24. Ehrenforth S, Kreuz W, Scharrer I, et al. Incidence of factor VIII and factor IX inhibitors in hemophiliacs. Lancet. 1992;339:594–598
  25. Lusher JM. Is the incidence and prevalence of inhibitors great with recombinant products? No. J Thromb Heamost. 2004;2:863–865
  26. Aledort LM. Is the incidence and prevalence of inhibitors greater with recombinant products? Yes. J Thromb Heaemost. 2004;2:861–862
  27. Gensana M, Altiesent C, Aznar JA, et al. Influence of von Willebrand factor on the reactivity of human factor VIII inhibitors. Haemophilia. 2001;7:369–374
  28. Behrmann M, Pasi J, Saint-Remy JMR, et al. Von Willebrand factor modulates factor VIII immunogenicity: comparative study of different factor VIII concentrates in a hemophilia A mouse model. Thromb Haemost. 2002;88:221–229
  29. Thompson AR. Structure, function, and molecular defects of factor IX. Blood. 1986;67:565–672
  30. Hershgold EJ, Pool JG, Pappenhagen AR. The potent antihemophilic globulin concentrate derived from a cold insoluble fraction of human plasma: characterization and further data on preparation and clinical trial. J Lab Clin Med. 1966;67:23–32
  31. Aldort LM. Introduction and overview of treatment. Semin Hematol. 1991;28:1–2
  32. Lusher JM. Thrombogenicity associated with factor IX complex concentrates. Semin Hematol. 1991;28:3–5
  33. Campbell EW, Neff S, Bowdler AJ. Therapy with factor IX concentrate resulting in DIC and thromboembolic phenomena. Transfusion. 1978;18:94–97
  34. Kasper CK. Postoperative thromboses in hemophilia B (letter). N Eng J Med. 1973;289:160
  35. Blatt PM, Lundblad RL, Kingdon HS, et al. Thrombogenic materials in prothrombin complex concentrates. Ann Intern Med. 1974;81:766–770
  36. Menache D, Behre HE, Orthner CL, et al. Coagulation factor IX concentrate: method of preparation and assessment of potential in vivo thrombogenicity in animal models. Blood. 1984;64:1220–1227
  37. Julius C, Westphal RG. The safety of blood components and derivatives. Hematol Oncol Clin North Am. 1992;6:1057–1077
  38. Mannucci PM, Bauer KA, Gringeri A, et al. Thrombin generation is not increased in the blood of hemophilia B patients aft the infusion of a purified factor IX concentrate. Blood. 1990;76:2540–2545
  39. Thompson AR. Factor IX concentrates for clinical use. Semin Thromb Hemost. 1993;19:25–36
  40. Berntorp E, Bjorkman S, Carlsson M, et al. Biochemical and in vivo properties of high purity factor IX concentrates. Thromb Haemost. 1993;70:768–773
  41. Abshire T, Shapiro A, Gill J et al. Recombinant FIX (rFIX) in the treatment of previously untreated patients with severe or moderately severe hemophilia B. Presented at XXIII International Congress of the World Federation of Haemophilia, The Hague, The Netherlands; 1998.
  42. Tabor E. The epidemiology of virus transmission by plasma derivatives: clinical studies verifying the lack of transmission of hepatitis B and C viruses and HIV type 1. Transfusion. 1999;39:1160–1168
  43. Future HK, Horowitz B, Bonomo R, et al. Solvent/detergent treated plasma: a virus substitute for fresh frozen plasma. Blood. 1992;79:826–831
  44. Hilgartner M, Aledort L, Andes A, Gill J. Efficacy and safety of vapor-heated anti-inhibitor complex in hemophilia patients. FEIBA Study Group. Transfusion. 1990;30:626–630
  45. Shapiro AD, Ragni MV, Lusher JM, et al. Safety and efficacy of monoclonal antibody purified factor IX concentrate in previously untreated patients with hemophilia B. Thromb Haemost. 1996;75:30
  46. Zauber NP, Levin J, et al. Factor IX level in patients with hemophilia B (Christmas disease) following transfusion with concentrates of factor IX fresh frozen plasma. Medicine. 1977;56:213–224
  47. Berntorp E, Bjorkman S, Carrlson M, et al. Biochemical and in vivo properties of high purity factor IX concentrates. Thromb Haemost. 1993;70:768–773
  48. Poon MC, Aledort LM, Anderle K, et al. Comparison of the recovery and half-life of a high purity factor IX concentrate with those of a factor IX complex concentrate. Factor IX Study Group. Transfusion. 1995;35:319–323
  49. Goldsmith JC, Kasper CK, Blatt PM, et al. Coagulation factor IX. Successful surgical experience with purified factor IX concentrate. Am J Hematol. 1992;40:210–215
  50. Hoots WK. The future of plasma-derived clotting factor concentrates. Haemophilia. 2001;7:4–9
  51. Kisker CT, Eisberg A, Schwartz B, Mononine Study Group . Prophylaxis in factor IX deficiency product and patient variation. Haemophilia. 2003;9:279–284
  52. Warrier I, Ewenstein BM, Koerper MA, et al. Factor IX inhibitors and anaphylaxis in hemophilia B. J Pediatr Hematol Oncol. 1997;19:23–27
  53. Mathew P, Altisent C. Recombinant versus plasma-derived factor IX in the treatment of hemophilia B: proceedings of a meeting held in June 2003. Blood Coagul Fibrin. 2004;15:S1–S3
  54. Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand disease. Blood. 1987;69:454–459
  55. Werner EJ, Boxson EH, Tucker EL, et al. Prevalence of von Willebrand disease in children: a multi-ethnic study. J Pediatr. 1993;123:893–898
  56. Scott JP, Montgomery RR. Therapy of von Willlebrand disease. Semin Thromb Hemost. 1993;19:37–47
  57. Weiss HJ, Sussman II, Hoyer LW. Stabilization of factor VIII in plasma by the von Willebrand factor: studies on posttransfusion and dissociated factor VIII and in patients with von Willebrand's Disease. J Clin Invest. 1977;60:390–404
  58. Manuucci PM. Treatment of von Willebrand's Disease. N Engl J Med 351;7:683–94.
  59. Sadler JE. A revised classification of von Willebrand disease. Thromb Haemost. 1994;71:520–525
  60. Holmberg L, Nilsson IM. von Willebrand's Disease. Eur J Haematol 192;48:127–41.
  61. Perkins HA. Correction of the hemostatic defects in von Willebrand's Disease. Blood. 1967;30:375–380
  62. Gill CJ, Ewenstein BM, Thompson AR, et al. Successful treatment of urgent bleeding in von Willebrand disease with FVIII/VWF concentrate (Humate-P): use of ristocetin factor assay (VWF:RCo) to measure potency and to guide therapy. Haemophilia. 2003;9:688–695
  63. Thompson AR, Gill JC, Ewenstein BM, et al. Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate-P). Haemophilia. 2004;10:42–51
  64. Mannucci PM, Chediak j, Hanna W, et al. Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study. Blood. 2002;99:450–456
  65. Kasper CK. Von Willebrand Disease Primer. Aventis Behring Foundation for Research and Advancement of Patient Health. 2004;p. 1–58
  66. Schimpf K, Brackmann HH, Kreuz W, et al. Absence of anti-human immunodeficiency virus type I and 2 seroconversion after the treatment of hemophilia and von Willebrand disease with pasteurized factor VIII concentrate. N Engl J Med. 1989;26(321):1148–1152
  67. Mazurier C, De Romeuf C, Parquet-Gernez A, Goudemand M. In vitro and in vivo characterization of a high-purity solvent/detergent-treated factor VIII concentrate: evidence for its therapeutic efficacy in von Willebrand disease. Eur J Haematol. 1989;43:7–14
  68. Turecek P, Gritsch H, Pichler L, et al. In vivo characterization of recombinant von Willebrand factor in dogs with von Willebrand disease. Blood. 1997;90:3555–3567
  69. Schimpf K, Mannucci PM, Kreutz W, et al. Absence of hepatitis after treatment with a pasteurized factor VIII concentrate in patients with hemophilia and no previous transfusions. N Eng J Med. 1987;316:918–922
  70. Mannucci PM, Chediak J, Hanna W, et al. Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study. Blood. 2002;99:450–456
  71. Hanna WT, Bona RD, Zimmerman CE, et al. The use of intermediate and high purity factor VIII products in the treatment of von Willebrand disease. Thromb Haemost. 1994;71:173–179
  72. Favaloro EJ, Bukuva M, Martinelli T, et al. A comparative mulit-laboratory assessment of three factor VIII/von Willebrand factor concentrates. Thromb Haemost. 2002;87:466–476
  73. Goudemand J, Mazurier C, Marey A, et al. Clinical and biological evaluation in von Willebrand's disease of a von Willebrand factor concentrate wit low factor VIII activity. Br J Haematol. 1992;80:21–221
  74. Goudemand J, Negrier C, Onnoughene N, Sultan Y. Clinical management of patients with von Willebrand's disease with VHP VWF concentrate: the French experience. Haemophilia. 1998;4:48–52
  75. Aventis Behring. Humate-P package insert; July, 2000.

PII: S1521-6926(05)00032-0

doi: 10.1016/j.beha.2005.01.031

Best Practice & Research Clinical Haematology
Volume 19, Issue 1 , Pages 35-49 , March 2006

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